Safety and Efficacy Data Published for Novel Nusinersen Drug Delivery Method for Spinal Muscular Atrophy Patients

A recently published paper details the safety and efficacy of nusinersen administration via a subcutaneous intrathecal catheter system (SIC) for SMA patients with advanced disease. SMA is a devastating genetic disease that leads to progressive degeneration of motor neurons that control movement, swallowing, and breathing. The novel SIC system is comprised of an intrathecal catheter that’s connected to an implantable infusion port. The SIC system was created to address the complications of nusinersen delivery derived from significant spinal deformities or fusions commonly found in SMA patients.

In the safety study, seventeen (17) participants started therapy between 2.7 and 31.5 years of age and received between 9 and 12 doses of nusinersen via SIC. A separate efficacy study included a subgroup of 11 participants with three copies of SMN2 who were nonambulatory, treatment-naïve, and had complex spine anatomy.

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